Literature DB >> 19508850

Finding the needle in the hay stack: hematopoietic stem cells in Fanconi anemia.

Lars U W Müller1, David A Williams.   

Abstract

Fanconi anemia is a rare bone marrow failure and cancer predisposition syndrome. Childhood onset of aplastic anemia is one of the hallmarks of this condition. Supportive therapy in the form of blood products, androgens, and hematopoietic growth factors may boost blood counts temporarily. However, allogeneic hematopoietic stem cell transplantation (HSCT) currently remains the only curative treatment option for the hematologic manifestations of Fanconi anemia (FA). Here we review current clinical and pre-clinical strategies for treating hematopoietic stem cell (HSC) failure, including the experience with mobilizing and collecting CD34+ hematopoietic stem and progenitor cells as target cells for somatic gene therapy, the current state of FA gene therapy trials, and future prospects for cell and gene therapy.

Entities:  

Mesh：

Substances：

Year:  2009        PMID： 19508850      PMCID： PMC2815349          DOI： 10.1016/j.mrfmmm.2009.03.010

Source DB:  PubMed          Journal:  Mutat Res        ISSN： 0027-5107            Impact factor:   2.433


  105 in total

1.  Transduction of nondividing cells using pseudotyped defective high-titer HIV type 1 particles.

Authors:  J Reiser; G Harmison; S Kluepfel-Stahl; R O Brady; S Karlsson; M Schubert
Journal:  Proc Natl Acad Sci U S A       Date:  1996-12-24       Impact factor: 11.205

2.  Germ cell defects and hematopoietic hypersensitivity to gamma-interferon in mice with a targeted disruption of the Fanconi anemia C gene.

Authors:  M A Whitney; G Royle; M J Low; M A Kelly; M K Axthelm; C Reifsteck; S Olson; R E Braun; M C Heinrich; R K Rathbun; G C Bagby; M Grompe
Journal:  Blood       Date:  1996-07-01       Impact factor: 22.113

3.  Prolonged administration of granulocyte colony-stimulating factor (filgrastim) to patients with Fanconi anemia: a pilot study.

Authors:  W R Rackoff; A Orazi; C A Robinson; R J Cooper; B P Alter; M H Freedman; R E Harris; D A Williams
Journal:  Blood       Date:  1996-09-01       Impact factor: 22.113

4.  gamma-Interferon gene expression in the bone marrow of patients with aplastic anemia.

Authors:  A Nisticò; N S Young
Journal:  Ann Intern Med       Date:  1994-03-15       Impact factor: 25.391

5.  In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector.

Authors:  L Naldini; U Blömer; P Gallay; D Ory; R Mulligan; F H Gage; I M Verma; D Trono
Journal:  Science       Date:  1996-04-12       Impact factor: 47.728

Review 6.  CD34+ selected cells in clinical transplantation.

Authors:  R H Collins
Journal:  Stem Cells       Date:  1994-11       Impact factor: 6.277

7.  Bone marrow transplantation for Fanconi anemia.

Authors:  E Gluckman; A D Auerbach; M M Horowitz; K A Sobocinski; R C Ash; M M Bortin; A Butturini; B M Camitta; R E Champlin; W Friedrich; R A Good; E C Gordon-Smith; R E Harris; J P Klein; J J Ortega; R Pasquini; N K Ramsay; B Speck; M R Vowels; M J Zhang; R P Gale
Journal:  Blood       Date:  1995-10-01       Impact factor: 22.113

8.  Abnormal lymphokine production: a novel feature of the genetic disease Fanconi anemia. II. In vitro and in vivo spontaneous overproduction of tumor necrosis factor alpha.

Authors:  F Rosselli; J Sanceau; E Gluckman; J Wietzerbin; E Moustacchi
Journal:  Blood       Date:  1994-03-01       Impact factor: 22.113

9.  Hematologic abnormalities in Fanconi anemia: an International Fanconi Anemia Registry study.

Authors:  A Butturini; R P Gale; P C Verlander; B Adler-Brecher; A P Gillio; A D Auerbach
Journal:  Blood       Date:  1994-09-01       Impact factor: 22.113

10.  The chemokine SDF-1 is a chemoattractant for human CD34+ hematopoietic progenitor cells and provides a new mechanism to explain the mobilization of CD34+ progenitors to peripheral blood.

Authors:  A Aiuti; I J Webb; C Bleul; T Springer; J C Gutierrez-Ramos
Journal:  J Exp Med       Date:  1997-01-06       Impact factor: 14.307
View more
  13 in total

Review 1.  Hematopoietic stem cell engineering at a crossroads.

Authors:  Isabelle Rivière; Cynthia E Dunbar; Michel Sadelain
Journal:  Blood       Date:  2011-11-17       Impact factor: 22.113

2.  Cytokinesis failure and attenuation: new findings in Fanconi anemia.

Authors:  Philip J Mason; Monica Bessler
Journal:  J Clin Invest       Date:  2010-12-22       Impact factor: 14.808

3.  Allogeneic hematopoietic cell transplantation for fanconi anemia in patients with pretransplantation cytogenetic abnormalities, myelodysplastic syndrome, or acute leukemia.

Authors:  Mouhab Ayas; Wael Saber; Stella M Davies; Richard E Harris; Gregory A Hale; Gerard Socie; Jennifer LeRademacher; Monica Thakar; H Joachim J Deeg; Amal Al-Seraihy; Minoo Battiwalla; Bruce M Camitta; Richard Olsson; Rajinder S Bajwa; Carmem M Bonfim; Ricardo Pasquini; Margaret L Macmillan; Biju George; Edward A Copelan; Baldeep Wirk; Abdullah Al Jefri; Anders L Fasth; Eva C Guinan; Biljana N Horn; Victor A Lewis; Shimon Slavin; Polina Stepensky; Marc Bierings; Robert Peter Gale
Journal:  J Clin Oncol       Date:  2013-04-01       Impact factor: 44.544

4.  Inherited DNA Repair Defects Disrupt the Structure and Function of Human Skin.

Authors:  Sonya Ruiz-Torres; Marion G Brusadelli; David P Witte; Kathryn A Wikenheiser-Brokamp; Sharon Sauter; Adam S Nelson; Mathieu Sertorio; Timothy M Chlon; Adam Lane; Parinda A Mehta; Kasiani C Myers; Mary C Bedard; Bidisha Pal; Dorothy M Supp; Paul F Lambert; Kakajan Komurov; Melinda Butsch Kovacic; Stella M Davies; Susanne I Wells
Journal:  Cell Stem Cell       Date:  2020-11-23       Impact factor: 24.633

5.  Second Allogeneic Hematopoietic Cell Transplantation for Patients with Fanconi Anemia and Bone Marrow Failure.

Authors:  Mouhab Ayas; Mary Eapen; Jennifer Le-Rademacher; Jeanette Carreras; Hisham Abdel-Azim; Blanche P Alter; Paolo Anderlini; Minoo Battiwalla; Marc Bierings; David K Buchbinder; Carmem Bonfim; Bruce M Camitta; Anders L Fasth; Robert Peter Gale; Michelle A Lee; Troy C Lund; Kasiani C Myers; Richard F Olsson; Kristin M Page; Tim D Prestidge; Mohamed Radhi; Ami J Shah; Kirk R Schultz; Baldeep Wirk; John E Wagner; H Joachim Deeg
Journal:  Biol Blood Marrow Transplant       Date:  2015-06-23       Impact factor: 5.742

6.  Fancd2-/- mice have hematopoietic defects that can be partially corrected by resveratrol.

Authors:  Qing-Shuo Zhang; Laura Marquez-Loza; Laura Eaton; Andrew W Duncan; Devorah C Goldman; Praveen Anur; Kevin Watanabe-Smith; R Keaney Rathbun; William H Fleming; Grover C Bagby; Markus Grompe
Journal:  Blood       Date:  2010-09-08       Impact factor: 22.113

7.  Loss of Faap20 Causes Hematopoietic Stem and Progenitor Cell Depletion in Mice Under Genotoxic Stress.

Authors:  Tingting Zhang; Andrew F Wilson; Abdullah Mahmood Ali; Satoshi H Namekawa; Paul R Andreassen; Amom Ruhikanta Meetei; Qishen Pang
Journal:  Stem Cells       Date:  2015-05-25       Impact factor: 6.277

8.  Formaldehyde catabolism is essential in cells deficient for the Fanconi anemia DNA-repair pathway.

Authors:  Ivan V Rosado; Frédéric Langevin; Gerry P Crossan; Minoru Takata; Ketan J Patel
Journal:  Nat Struct Mol Biol       Date:  2011-11-13       Impact factor: 15.369

Review 9.  Yin and Yang: The dual effects of interferons on hematopoiesis.

Authors:  Yasmin Demerdash; Bailee Kain; Marieke A G Essers; Katherine Y King
Journal:  Exp Hematol       Date:  2021-02-08       Impact factor: 3.084

10.  Phenotypic correction of Fanconi anemia cells in the murine bone marrow after carrier cell mediated delivery of lentiviral vector.

Authors:  Santhosh Chakkaramakkil Verghese; Natalya A Goloviznina; Peter Kurre
Journal:  Stem Cell Res Ther       Date:  2016-11-19       Impact factor: 6.832
View more

北京卡尤迪生物科技股份有限公司 © 2022-2023.